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J Pediatr Gastroenterol Nutr. 2019 Jan 17. doi: 10.1097/MPG.0000000000002256. [Epub ahead of print]

A Phase I/IIa Trial of Intravenous Immunoglobulin Following Portoenterostomy in Biliary Atresia.

Author information

1
Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO.
2
University of Michigan, Ann Arbor, MI.
3
Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL.
4
Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
5
Children's Hospital Los Angeles, Los Angeles, CA.
6
The Hospital for Sick Children, University of Toronto, Toronto, Canada.
7
Emory University School of Medicine, Atlanta, GA.
8
Children's Hospital of Pittsburgh, Pittsburgh, PA.
9
Children's Hospital of Philadelphia, Philadelphia, PA.
10
National Institute of Diabetes, Digestive and Kidney Diseases, NIH, Bethesda, MA.

Abstract

OBJECTIVES:

Biliary atresia (BA) is a progressive neonatal fibroinflammatory cholangiopathy. We hypothesized that intravenous immunoglobulin (IVIg) would be safe, feasible, acceptable and efficacious for the treatment of BA. The primary objective of this study was to establish the feasibility, acceptability and safety profile of IVIg administration after hepatoportoenterostomy (HPE) in BA. The secondary objective was to determine the treatment efficacy of IVIg based on good bile drainage and survival with the native liver.

METHODS:

A multi-center, prospective, open-labeled, phase I/IIA trial of IVIg was conducted, with 1 gm/kg/dose of IVIg infused at 3-5 days, 30 days and 60 days post-HPE, and subjects followed for 360 days post-HPE. Twenty-nine participants completed the study.

RESULTS:

Administration of IVIg infusions was feasible and acceptable in 79%. None of the serious adverse events (SAEs) were directly related to IVIg infusions, however 90% of participants had an SAE. Compared to a historical placebo-arm group, there was no significant increase in the proportion of IVIg participants with a serum total bilirubin < 1.5 mg/dL at 90, 180, or 360 days post-HPE. Survival with the native liver in the IVIg participants showed no significant benefit over the historical placebo-arm, with a difference at 360 days of -11.9% (IVIg:58.6%, placebo:70.5%; 90% UCB:2.1%; p > 0.05).

CONCLUSIONS:

Although IVIg infusions in infants with BA post-HPE were feasible, acceptable and safe, there was no trend to lower bilirubin levels or improved 360 day survival with the native liver.

CLINICAL TRIAL:

Safety Study of Intravenous Immunoglobulin Post-Portoenterostomy in Biliary Atresia; #NCT01854827.

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